Solid Biosciences Announces Licensing Agreement with Kinea Bio for the Use of Proprietary Next-Generation Capsid AAV-SLB101
- Non-exclusive license for Solid’s proprietary, next generation capsid, AAV-SLB101, to accelerate development of Kinea Bio’s gene therapy for dysferlin-related limb-girdle muscular dystrophy -
- Solid to receive an upfront payment and is eligible for certain development and sales milestones and tiered royalties on net sales -
- Solid continues to expand collaborative efforts for AAV-SLB101 with more than 25 agreements and licenses executed to date -
CHARLESTOWN, Mass., Sept. 23, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced a non-exclusive worldwide license and collaboration agreement with Kinea Bio for the use of Solid’s proprietary, next-generation capsid, AAV-SLB101, for the development and commercialization of KNA-155, an investigational dual AAV gene therapy targeting dysferlinopathy, a form of limb-girdle muscular dystrophy type 2B/R2 (LGMD2B/R2).
Under the terms of the agreement, Solid granted Kinea Bio a non-exclusive worldwide license to utilize AAV-SLB101 as the delivery backbone for KNA-155, which is advancing into IND-enabling preclinical activities. In return, Solid receives an upfront fee and is eligible for additional payments upon the achievement of certain development and sales milestones and tiered royalties on net sales.
AAV-SLB101 is Solid’s rationally designed capsid developed for enhanced muscle tropism and reduced biodistribution to the liver. Robust cardiac and skeletal muscle transduction and biodistribution have been demonstrated in preclinical studies as well as in early clinical data from Solid’s ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial (NCT06138639) evaluating SGT-003 (which utilizes AAV-SLB101), an investigational gene therapy to treat Duchenne muscular dystrophy. As of a data cutoff of August 12, 2025, AAV-SLB101 has been well tolerated in the 15 participants who have been dosed in the INPSIRE DUCHENNE trial.
“We are pleased to partner with the Kinea Bio team to expand AAV-SLB101’s application into dysferlin-related LGMD,” said Bo Cumbo, President and CEO of Solid Biosciences. “As we progress the INSPIRE DUCHENNE trial, the first-in-human evaluation of this next-generation capsid, we remain highly encouraged by the emerging safety profile, which we believe continues to de-risk and build the value proposition of AAV-SLB101 as a differentiated, muscle tropic capsid.
“At Solid, we are wholly focused on shaping a brighter future for gene therapy by creating the next wave of delivery tools, including capsids, promoters and CMC technology, all of which are developing into a rich constellation of synergistic tools that we believe will power future generations of gene therapies for patients,” Mr. Cumbo concluded.
“Solid’s next-generation capsid, AAV-SLB101, contributes critical, cutting-edge technology that we believe will provide a powerful delivery mechanism for our KNA-155 program,” said Casey Childers, CEO of Kinea Bio. “The robust preclinical and clinical data generated to date for AAV-SLB101 give us confidence in our potential to bring a safe and effective therapy to patients living with dysferlinopathies, and we look forward to partnering with the highly experienced team at Solid as we advance development.”
About AAV-SLB101
AAV-SLB101 is a proprietary, rationally designed capsid developed for enhanced muscle tropism and reduced liver uptake. With a robust preclinical package in mice and nonhuman primates, AAV-SLB101 has demonstrated increased transduction speed, enhanced skeletal and cardiac muscle tropism, decreased liver biodistribution and improved efficiency when compared to first generation capsids. The incorporation of AAV-SLB101 into AAV delivered therapies has the potential to be a step forward in the treatment of neuromuscular and cardiac diseases. Solid Biosciences aims to license AAV-SLB101 broadly to both companies and academic institutions pursuing treatments for rare diseases. Solid has existing licensing agreements with more than 25 academic labs, institutions, and companies for the use of AAV-SLB101.
About Solid Biosciences
Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy (Duchenne), SGT-212 for Friedreich’s ataxia (FA), SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), SGT-601 for TNNT2-mediated dilated cardiomyopathy and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com.
About Kinea Bio, Inc.
Kinea Bio, Inc. is a biotechnology company pioneering a novel dual AAV vector platform SIMPLI-GTTM to deliver large therapeutic genes that exceed the natural packaging capacity of AAV. The company was among the first to demonstrate the potential of this approach in systemic disorders such as Duchenne muscular dystrophy and is now expanding its pipeline to include dysferlinopathy (LGMD2B/R2) and other severe genetic conditions. Through innovative science and strategic collaborations, Kinea Bio is dedicated to translating breakthrough biology into transformative medicines for patients with high unmet needs. For more information, please visit www.kineabio.com.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the Company; the ability to successfully achieve and execute on the company’s goals, priorities and achieve key clinical milestones; the Company’s pipeline of capsid products, including SLB-101, and programs for neuromuscular and cardiac diseases, including its SGT-501, SGT-212 and SGT-003 programs and expectations for CTA filings, site activations, clinical development, initiation and enrollment in clinical trials, dosing, availability of clinical trial data and potential accelerated approval; the sufficiency of the Company’s cash, cash equivalents, and available-for-sale securities to fund its operations; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the company’s ability to advance and license AAV-SLB101 and advance SGT-212, SGT-003, SGT-501, SGT-601, SGT-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the company’s product candidates; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne, Friedreich’s ataxia and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-212, SGT-003, SGT-501, SGT-601, SGT-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the company’s views as of the date hereof and should not be relied upon as representing the company’s views as of any date subsequent to the date hereof. The company anticipates that subsequent events and developments will cause the company's views to change. However, while the company may elect to update these forward-looking statements at some point in the future, the company specifically disclaims any obligation to do so.
Solid Biosciences Investor Contact:
Nicole Anderson
Director, Investor Relations and Corporate Communications
Solid Biosciences Inc.
investors@solidbio.com
Media Contact:
Glenn Silver
FINN Partners
glenn.silver@finnpartners.com
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